This is a pivotal year for Ionis Pharmaceuticals, a pioneer in RNA therapeutics.
After moving from a partnership model to a wholly owned strategy following Tringorza’s 2024 approval, the company is counting on the FDA’s decision to expand the drug into severe hypertriglyceridemia as its first non-orphan indication for a wholly owned product. Meanwhile, potentially game-changing Phase 3 results are expected from Ionis’ lipoprotein(a) candidate in partnership with Novartis and the ATTR cardiomyopathy program in partnership with AstraZeneca.
On this week’s episode of The Topline, Fierce’s Angus Lew interviews Dr. Brett Monier, CEO of Ionis. He talks about the science of antisense oligonucleotides, the importance of two upcoming clinical results, and what it will take to turn a 30-year-old discovery platform into a full-fledged commercial biotechnology.

